ReachMDPromising Results in Trial of First “Gene Silencing” Drug for Alzheimer’s Disease

More trials in larger groups of patients will be required to determine whether this drug, BIIB080 (/IONIS-MAPTRx), an antisense oligonucleotide (used to stop RNA from producing a protein), leads to clinical benefit, but the phase 1 results published in Nature Medicine – with results from 46 patients – are the first indication that this method has a biological effect.

BIIB080 ‘silences’ the tau protein-coding gene, known as the microtubule-associated protein tau (MAPT) gene. This inhibits the gene from being dosed and reversibly translated into protein. It will also reduce the creation of that protein, changing the course of the condition.